ALS Prediction Prize

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THE NEXT ALS BREAKTHROUGH COULD BE YOURS.

Big Data in Medicine: Prize4Life ALS Prediction Challenge

The DREAM-Phil Bowen

ALS Prediction Prize

Bruce TomanCornell University, on behalf of

Prize4Life and פרס לחיים

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Medicine

• Technology = applied science• IT

– Semiconductor = applied physics– Encryption algorithms = applied math

• Medicine– applied biology… but… http://www.youtube.com/watch?v=RrS2uROUjK4&feature=BFa&list=PL757C8E14C021A1BF

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Big Data in Medicine• Clinical trial vs basic science data• Basic science: gene sequences, protein

structures, small molecule structures, etc• Clinical data

– Patient data• Corporate owned: EHRs- huge privacy issues• New “open source” databases – 23andMe,

PatientsLikeMe, Sage Bionetworks

– Clinical trial data• Corporate owned• Consortia: CAMD Alzheimer's disease database, and…

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Collaboration with the Northeast ALS Consortium (NEALS) and the Neurology Clinical Trials Unit at Massachusetts General Hospital

Will have > 8000 de-identified patient data records from 13 completed public and industry clinical trials - Largest merged ALS patient record data set ever

Invaluable resource to help address questions around ALS natural history, trial design, patient stratification, and biomarkers

Will be made available in December 2012

The Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) Project

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What is ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s Disease)?

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Lou Gehrig (died within 2 years of diagnosis)

Stephen Hawking (has lived with the disease for over 40 years)

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The only existing FDA-approved treatment, approved in 1995, prolongs life by only 2-3 months

Orphan disease - 30k in the US (vs. >25 million diabetes patients)

Challenge of ALS clinical trialsDisease progression varies, so need many patients for

statistical powerLimited number of patients available, so takes a long

timeHigh expense of these trials drives companies away

Challenges for ALS Therapy Development

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Prize4Life (P4L) Aims to Accelerate Therapy Development for ALS

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P4L is a results-oriented non-profit organization focused exclusively on ALS

Founded in 2006 by Avichai Kremer and Harvard Business School colleagues when he was diagnosed at age 29.

P4L adopted the Incentive Prize Model Focus research on unmet needs in ALS Lower risk for therapy-development companies Bring new ideas and new minds into the field

Avi, ~9 mo after diagnosis

winner of the 2011 PM award for Entrepreneurship and Innovation~7 years after diagnosis

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Incentive Prizes Accelerate Scientific Innovation

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1927: The Orteig Prize revolutionized modern aviation (Charles Lindbergh)

2004: The Ansari X-Prize revolutionized personal space flight. (Spaceship One)

2005: The DARPA Grand Challenge - robotics

2012: The Archon Genomics X Prize: Energizing personal medicine

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The DREAM-Phil Bowen ALS Prediction Prize

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$25,000 for a Predictive Algorithm of ALS Disease ProgressionJuly 15 - October 15, 2012

www.innocentive.com/ar/challenge/9933047

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The Prize has Launched!Winner gets $25,000Speaking invitation and travel expenses to the

DREAM7 conference (Nov 12-15, San Francisco) for one member of the three best performing teams.

Make an impact for ALS patients reducing clinical trial costs => more drugs tested!

Solve an intriguing challenge

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The challenge details• The training data contains 900 patients tracked for 12 months or more• For a given patient, between 100 and ~200 datapoints. ~135,000 datapoints.• Goal: Based on the information from the first 3 months , predict the progression of

the disease over the next 9 months • Progression- a change a scale called ALSFRS (ALS functional rating scale) ranging

from 0-40– 10 questions with a score of 0-4 for each one, with4 being normal, 0 being complete inability)– Predict (ALSFRS(12)-ASFRS(3))/# of months

• Data: demographics, family history, medications, symptoms, lab results(blood and urine), all available over a year’s time

• Data is spotty. This is reality of clinical trials. One of the great things in this challenge. But your algorithm needs to deal with this.

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Submitting Your Solution July 15 - October 1:

Develop your code with a training set Test your code on a leaderboard set that you cannot see. Innocentive

provides your score and ranks you on a leaderboard On October 1: solvers obtain the leaderboard set to further

improve their algorithms By October 15: Submit final code in R and a description Algorithms will be evaluated with a validation set. A winner will be announced in early November.

Judges include Merit Cudkowitz,MD Director, Neurology Clinical Trials Unit, MGH; Gustavo Stolovitzy, PhD – IBM Computational Biology Center, co-Founder of DREAM

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The Winner!

The solver with an algorithm with smallest RMSD and a complete submission will win!

The solver is required to publish their algorithm description (alone or in coordination with Prize4Life)

Solver retains all intellectual property rights in the code.

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Join the Competition!

We have the data, the only thing missing is you!

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THE NEXT ALS BREAKTHROUGH COULD BE YOURS